Selective Class II HDAC Inhibitors for Autoimmune Disease Treatment

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Solution Overview

Problem

Current HDAC inhibitors are non-selective, affecting all HDAC isoforms, which can lead to unwanted side effects and reduced efficacy in treating specific diseases associated with abnormal cell proliferation and immune regulation.

Innovation Solution

Development of a compound according to Formula (Ia) and (Ib) that selectively inhibits Class II HDAC activity, specifically targeting HDAC9 or HDAC7 or HDAC6, to modulate autoimmune diseases and enhance Treg activity.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If non-selective HDAC inhibitors are used, then HDAC activity is inhibited across all isoforms, but unwanted side effects increase and efficacy for specific diseases is reduced

Engineering Contradiction:
Improveefficacy for specific diseasesVSAvoidunwanted side effects
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies local quality by designing HDAC inhibitors with specific molecular structures ( Formula I compounds) that target particular HDAC isoforms (Class II HDACs: HDAC4, HDAC5, HDAC6, HDAC7, HDAC9) rather than inhibiting all HDAC isoforms non-selectively. This selective inhibition allows beneficial effects in specific disease contexts while avoiding harmful side effects associated with broad-spectrum inhibition.

Inventive Principle:
Principle #3Local quality

2Reliability

If selective Class II HDAC inhibitors are developed, then disease-specific efficacy is improved, but compound complexity increases

Engineering Contradiction:
Improvedisease-specific efficacyVSAvoidcompound structure complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The patent employs parameter changes by systematically varying molecular parameters (substituents R1-R6, n values, Z groups) in the core scaffold structure to optimize selectivity for Class II HDACs. By adjusting these parameters, the compounds achieve disease-specific efficacy while maintaining manageable structural complexity through a unified chemical series approach.

Inventive Principle:
Principle #35Parameter changes

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The selective inhibition of Class II HDACs, such as HDAC9 or HDAC7 or HDAC6, enhances Treg suppression and expansion, potentially treating autoimmune diseases, cancer, and other conditions like colitis and cardiac allograft survival with reduced side effects.

Implementation Method 1

HDAC inhibitors and their use in the treatment of cellular proliferative diseases, including cancer, neurodegenerative diseases, schizophrenia and stroke

Methodology Applied
Scientific EffectEnzyme inhibition: Enzyme

Implementation Method 2

Histone deacetylases (HDACs) catalyze the deacetylation of histone and non-histone proteins

Methodology Applied
Scientific EffectDeacetylation: Hydrolysis

Data Source

PatentEP2533783B8COMPOUNDS AND METHODS for the inhibition of HDAC
Publication Date: 2015.12.16 TEMPERO PHARMACEUTICALS INC

AI summary

Disclosed are compounds having the formula: wherein X1, X2, X3, R1, R2, R3, R4, Y, A, Z, L and n are as defined herein, and methods of making and using the same.